Conferences

Assessing Lentiviral Cell Therapy for Cystinosis: Stephanie Cherqui, PhD

March 14th 2022

The associate professor from UC San Diego discussed the ongoing phase ½ trial of CTNS-RD

Performing In-Depth Analysis of Lentiviral Cell Therapies: Luca Biasco, PhD

March 11th 2022

The director of research and development at AVROBIO discussed the molecular follow-up of cell therapies.

Cautious Optimism for GM1 Gangliosidosis Gene Therapy: David Weinstein, MD

March 10th 2022

The senior vice president of clinical development at Passage Bio discussed biomarker activity seen in the IMAGINE-1 trial.

Patient-Informed Study to Evaluate Hunter Syndrome Gene Therapy

February 23rd 2022

The trial design was informed by both patient and physician perspectives.

Lysogene Gene Therapies Show Promise in GM1 Gangliosidosis and MPSIIIA

February 15th 2022

Updated data were presented on LYS-SAF302 and LYS-GM101 at WORLDSymposium.

The Need for Analysis in Lentiviral Cell Therapies: Luca Biasco, PhD

February 15th 2022

The director of research and development at AVROBIO discussed the analytical pipeline the company has developed for molecular follow-up of cell therapies.

The Potential Impact of Gene and Cell Therapy in Cystinosis: Stephanie Cherqui, PhD

February 14th 2022

The associate professor from UC San Diego discussed the curative potential of gene therapy in cystinosis.

First In-Human Trial to Assess Krabbe Disease Gene Therapy

February 14th 2022

Passage Bio presented data on both the Krabbe and GM1 gangliosidosis programs at WORLDSymposium.

Gene Therapy Well-Tolerated in Late-Onset Pompe Disease

February 13th 2022

Interim safety data from the FORTIS study were presented at WORLDSympsoium.

Achieving Developmental Milestones With GM1 Gangliosidosis Gene Therapy: David Weinstein, MD

February 11th 2022

The senior vice president of clinical development at Passage Bio discussed new data from the IMAGINE-1 trial presented at WORLDSymposium.